CHANGES IN NEUROMUSCULAR JUNCTIONS IN ALS

ALS 神经肌肉接头的变化

• 批准号: 8167048
• 负责人: JAMES B CARESS
• 金额: $ 1.67万
• 依托单位: WAKE FOREST UNIVERSITY HEALTH SCIENCES
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-03-01 至 2011-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8167048
• 关键词: Blinded; Canada; Cessation of life; Clinical Trials; Computer Retrieval of Information on Scientific Projects Database; Controlled Clinical Trials; Disease Progression; Double-Blind Method; Drops; Funding; Grant; Institution; Italy; Lithium; Measures; Neuromuscular Junction; Outcome Measure; Participant; Pilot Projects; Placebo Control; Placebos; Randomized; Recruitment Activity; Research; Research Personnel; Resources; Riluzole; Safety; Serum; Site; Source; Time; Treatment Failure; United States National Institutes of Health; Visit; functional decline; primary outcome; volunteer

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. A small pilot study was performed in Italy in participants with ALS. Sixteen participants were treated with 300 ¿?? 450mg/day of lithium (target serum level 0.4-0.8 mEq/L) and riluzole; 28 participants received only riluzole. Participants treated with lithium demonstrated a slower rate of functional decline as measured by the ALSFRS-R. The study was small and participants were not blinded to treatment. This is a double blind, randomized, placebo-controlled clinical trial evaluating the safety and efficacy of lithium and riluzole compared to placebo and riluzole. Approximately 250 volunteers will be recruited from multiple centers in the US and Canada that are part of the Northeast ALS Consortium (NEALS) and the Canadian ALS Clinical Trials and Research Network (CALS). The primary outcome measure is disease progression defined as the time to decline of 6 points in the ALSFRS-R scale or death. The ALSFRS-R will be administered at the Baseline Visit; this will be considered the baseline score for each participant. A drop of 6 points or more in the ALSFRS-R will be defined as disease progression and treatment failure. Participants will then be treated with lithium for the period of the study (up to 52 weeks total). Study site staff will remain blinded to initial group assignments and will remain blinded to the total ALSFRS-R scores and be unaware of the date when a participant is switched from placebo to lithium.

该子项目是利用该技术的众多研究子项目之一 资源由 NIH/NCRR 资助的中心拨款提供。 研究者 (PI) 可能已从 NIH 的另一个来源获得主要资金， 因此可以出现在其他 CRISP 条目中 列出的机构是。 中心，不一定是研究者的机构。 在意大利对 ALS 参与者进行了一项小型试点研究，16 名参与者接受了 300 ¿ ?? 450 毫克/天的锂（目标血清水平 0.4-0.8 mEq/L）和利鲁唑；根据 ALSFRS-R 的测量，28 名参与者仅接受利鲁唑治疗，其功能下降速度较慢。参与者并未对治疗视而不见。 这是一项双盲、随机、安慰剂对照临床试验，评估锂和利鲁唑与安慰剂和利鲁唑相比的安全性和有效性。 将从美国和加拿大的多个中心招募约 250 名志愿者，这些中心属于东北 ALS 联盟 (NEALS) 和加拿大 ALS 临床试验和研究网络 (CALS)。 主要结果指标是疾病进展，定义为 ALSFRS-R 量表下降 6 分或死亡的时间。 ALSFRS-R 将在基线访视时进行；这将被视为每个参与者的基线评分下降。 ALSFRS-R 中 6 分或以上将被定义为疾病进展和治疗失败，然后参与者将在研究期间（总共 52 周）接受锂治疗。作业和将仍然对 ALSFRS-R 总分不知情，并且不知道参与者从安慰剂切换到锂盐的日期。